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PPMD 2024: 4 therapies aiming to slow DMD progression now in trials
Four experimental treatments — deramiocel, sevasemten, satralizumab, and tadalafil — all in clinical testing and all aiming to slow the progression of Duchenne muscular dystrophy (DMD), were spotlighted in a session at last week’s Parent Project Muscular Dystrophy (PPMD) conference. In Duchenne MD, mutations lead to a lack of functional dystrophin, a protein that normally…
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A rough month makes me grateful for what I’ve got
Last month was tough for me. Although celebrating my partner’s birthday on June 12 was a welcome distraction, I still had a June I’d like to forget, mainly because I spent the entire month recovering from a foot infection that hospitalized me for the last three days of May. And after that I had an…
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Seminario Web Cure Duchenne: Explicación de la Decisión de la FDA acerca de Elevidys
Watch HERE The post Seminario Web Cure Duchenne: Explicación de la Decisión de la FDA acerca de Elevidys appeared first on CureDuchenne.
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FDA APPROVAL OF ELEVIDYS
This webinar discusses a groundbreaking gene therapy treatment for Duchenne Muscular Dystrophy (DMD), focusing on a method to introduce a miniaturized version of the dystrophin gene using a viral vector. This therapy, while not a cure, aims to improve the quality of life for patients. The treatment involves a single dose, but patients must undergo…
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Dyne Therapeutics of DYNE-251 in Duchenne
Dyne Therapeutics shares with the community recent efficacy and safety data from the ongoing DELIVER trial of DYNE-251 in Duchenne. Watch HERE The post Dyne Therapeutics of DYNE-251 in Duchenne appeared first on CureDuchenne.
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ITF THERAPEUTICS| DUVYZAT Update
CureDuchenne Webinar | ITF THERAPEUTICS Patient Community Update: An overview of DUVYZAT Recorded on June 26, 2024Watch HERE The post ITF THERAPEUTICS| DUVYZAT Update appeared first on CureDuchenne.
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PPMD 2024: Building community is vital for mental, physical health
Connecting with a supportive community is essential for people affected by muscular dystrophy to maintain both their emotional and physical well-being, according to Brandon Kozar, a psychologist at Nationwide Children’s Hospital. “When we come together … something amazing happens. We become more powerful. We become connected. At the very least, we know that we’re not alone,”…
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PPMD 2024: PTC to seek FDA approval of ataluren for DMD
PTC Therapeutics plans to soon ask the U.S. Food and Drug Administration (FDA) to approve ataluren as a treatment for Duchenne muscular dystrophy (DMD) caused by nonsense mutations. “We are thrilled to announce that we are moving forward with the submission of a new drug application for ataluren here in the U.S.,” Jonathan Blaize, PhD,…
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PPMD 2024: DMD therapies to boost dystrophin advance in trials
More than half a dozen exon-skipping therapies for Duchenne muscular dystrophy (DMD) — all seeking to increase production of the dystrophin protein by modulating how the DMD gene is read — are now being explored in clinical trials, according to researchers. Updates on several of these investigational DMD treatments were discussed during a session at…
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Protected: Duchenne Bone Health Study
This content is password protected. To view it please enter your password below: Password: The post Protected: Duchenne Bone Health Study appeared first on CureDuchenne.
